← Back to Search

Gene Editing

CTX001 for Sickle Cell Disease and Thalassemia

Phase 3
Recruiting
Research Sponsored by Vertex Pharmaceuticals Incorporated
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up within 42 days after ctx001 infusion
Awards & highlights

Study Summary

This trial will test a new way to treat transfusion-dependent beta thalassemia or severe sickle cell disease, by using the patient's own modified stem cells. The goal is to see if this is safe and effective.

Who is the study for?
This trial is for individuals with transfusion-dependent β-thalassemia or severe sickle cell disease who are eligible for a stem cell transplant. It's not open to those with prior transplants, available matched donors, certain genetic conditions like α-thalassemia in TDT patients, or untreated moyamoya syndrome in SCD patients.Check my eligibility
What is being tested?
The study tests CTX001, which involves editing the patient's own stem cells using CRISPR-Cas9 technology and then returning them to the body. The goal is to see if this single-dose treatment can safely improve symptoms of these blood disorders.See study design
What are the potential side effects?
Potential side effects may include typical risks associated with stem cell transplants such as infection risk due to immune suppression during conditioning, possible infusion reactions, and complications from the gene-editing process.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~within 42 days after ctx001 infusion
This trial's timeline: 3 weeks for screening, Varies for treatment, and within 42 days after ctx001 infusion for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Fetal Hemoglobin
Total Hemoglobin (Hb) Concentration Over Time
Secondary outcome measures
SCD: Relative Reduction in Annualized Duration of Hospitalization for Severe VOCs
SCD: Relative Reduction in Annualized Rate of Inpatient Hospitalizations for Severe VOCs
SCD: Relative Reduction in Annualized Rate of Severe Vaso-Occlusive Crises (VOCs)
+14 more

Trial Design

1Treatment groups
Experimental Treatment
Group I: CTX001Experimental Treatment1 Intervention
CTX001 (autologous CD34+ hHSPCs modified with CRISPR-Cas9 at the erythroid lineage-specific enhancer of the BCL11A gene). Participants will receive a single infusion of CTX001 through a central venous catheter.

Find a Location

Who is running the clinical trial?

Vertex Pharmaceuticals IncorporatedLead Sponsor
243 Previous Clinical Trials
32,361 Total Patients Enrolled
CRISPR TherapeuticsIndustry Sponsor
5 Previous Clinical Trials
280 Total Patients Enrolled

Media Library

CTX001 (Gene Editing) Clinical Trial Eligibility Overview. Trial Name: NCT05477563 — Phase 3
Hemoglobin Disorders Research Study Groups: CTX001
Hemoglobin Disorders Clinical Trial 2023: CTX001 Highlights & Side Effects. Trial Name: NCT05477563 — Phase 3
CTX001 (Gene Editing) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05477563 — Phase 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Does the age restriction for this trial top out at 75 years old?

"This study is only for patients aged 12 to 35. There are currently 213 clinical trials underway for patients younger than 18 and 177 trials for patients older than 65."

Answered by AI

Can people still join this particular clinical trial?

"Correct, the clinical trial is currently ongoing and looking for 12 more patients to test the experimental intervention. The study was posted on 8/2/2022 and last edited on 11/10/2022."

Answered by AI

What are some of the short and long term risks associated with taking CTX001?

"CTX001 falls into the Phase 3 category on our safety scale, which means that there is some evidence of its efficacy and multiple rounds of data supporting its safety."

Answered by AI

What are the prerequisites for participants in this research project?

"The requirements for potential participants of this trial are that they must have a hematologic disease, be between 12 and 35 years old. There can only be a total of 12 people in the study."

Answered by AI

Who else is applying?

What portion of applicants met pre-screening criteria?
Did not meet criteria

Why did patients apply to this trial?

I've tried 3 other drugs that didn't work and now i'm hoping this trial will lead to a cure for my condition.
PatientReceived 2+ prior treatments
Recent research and studies
clinicaltrials.govStudy
Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
2022. "Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05477563.
All > Sickle Cell Disease > Sickle Cell
~8 spots leftby Feb 2025
Unbiased ResultsWe believe in providing patients with all the options.
Your Data Stays Your DataWe only share your information with the clinical trials you're trying to access.
Verified Trials OnlyAll of our trials are run by licensed doctors, researchers, and healthcare companies.
Back to top